Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease. A portion of cord blood cells (ALD-101) will be separated from the cord blood unit and given approximately 4 hours after the standard cord blood transfusion.

The study will test if the supplemental cells will increase the speed at which normal levels of circulating blood cells are re-established after transplant.

To assess the efficacy of adjuvant therapy of ALD-101 in accelerating platelet engraftment in patients also receiving a standard unrelated UCBT for treatment of inherited metabolic diseases [ Time Frame: 180 Days ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To assess the efficacy of ALD-101 in accelerating neutrophil engraftment [ Time Frame: 180 Days ] [ Designated as safety issue: No ]

A subpopulation of cord blood cells composed of cells that express a high level of the intracellular enzyme aldehyde dehydrogenase (ALDH).

Detailed Description:

Subjects will be hospitalized and undergo high doses of chemotherapy which will destroy the child's normal cells including their bone marrow (which forms blood cells) in order to prepare their body for the umbilical cord blood transplant. The cord blood transplant is intended to rescue your child's bone marrow from the bad effects of the procedure. The child will receive 80% of a standard cord blood transplant followed by 20% supplemental stem cell called ALD-101.

The study will evaluate if these cells (ALD-101) will repopulate the bone marrow more rapidly after transplant. This would shorten the period of time that the transplanted subject would be at risk for infection and bleeding and would also decrease the number of red blood cell and platelet transfusions needed.

ALD-101 has been used as a supplement to cord blood transplant in twenty-eight children with genetic diseases and malignancy in one previous clinical study that is on-going.

The main purpose of this research study is to test whether a portion of cord blood cells that have been separated from a cord blood unit (ALD-101) will increase the speed at which normal levels of circulating blood cells are re-established after transplant. This is the experimental part of this study. ALD-101 is also being tested to see if it is safe.

Eligibility

Ages Eligible for Study:

up to 16 Years

Genders Eligible for Study:

Both

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

confirmed diagnosis of inherited metabolic diseases; including the following:

Hurler Syndrome (MPS I)

Hurler-Scheie Syndrome

Hunter Syndrome (MPS II)

Sanfilippo Syndrome A and B(MPS III)

Maroteaux-Lamy Syndrome (MPS VI)

Krabbe Disease (Globoid Leukodystrophy)

Metachromatic Leukodystrophy (MLD)

Adrenoleukodystrophy (ALD and AMN)

Sandhoff Disease

Tay Sachs Disease

Pelizaeus Merzbacher (PMD)

Niemann-Pick Disease

Alpha-mannosidosis

I-Cell Disease (ML II)

Fucosidosis

GM I Gangliosidosis

Canavan Disease

must be <16 years of age at the time of study enrollment

must have a good performance status (Lansky ≥80%)

must have adequate function of other organ systems including: kidney, liver, heart and lungs

must have given valid written informed consent

must have a minimum life expectancy of at least 6 months

must be determined to be a good candidate for a standard umbilical cord blood transplant

must have an IQ >70 or if too young for IQ testing the potential to reach this endpoint by age 5

Exclusion Criteria:

HIV, Hepatitis B and/or Hepatitis C positive

concurrently involved in any other clinical study that affects engraftment or immune reconstitution

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00654433